NUZ-001 OLE Study Shows Promising Long-Term Safety and Efficacy in ALS.

Neurizon Therapeutics Limited (ASX: NUZ) has announced positive topline results from the Open-Label Extension (OLE) study of its lead candidate, NUZ-001, for amyotrophic lateral sclerosis (ALS). The study confirmed that long-term treatment at the recommended Phase 2 dose was safe and well-tolerated, with no treatment-related severe adverse events or discontinuations. Importantly, NUZ-001 demonstrated durable functional benefits, minimal respiratory decline, and encouraging biomarker trends, including a 17% reduction in urinary p75ECD.

Compared with matched historical controls from the PRO-ACT database, NUZ-001 significantly improved survival, reducing the risk of death by 76.7% and extending median survival by approximately 16 months. Functional decline slowed by 31%, while respiratory decline was reduced by 43%. With over 2.5 years of safe use under the TGA’s Special Access Scheme, five patients remain on treatment. These promising findings provide strong support for advancing NUZ-001 into the pivotal HEALEY ALS Platform Trial, expected to commence in Q4 CY2025.

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